Atsena Therapeutics has received orphan designation from the European Medicines Agency for its two clinical‑stage gene therapy candidates, ATSN‑101 for Leber congenital amaurosis 1 and ATSN‑201 for X‑linked retinoschisis. The company said the decision provides development incentives and market exclusivity in the EU as both programmes advance through global pivotal trials.
Patrick Ritschel, Chief Executive Officer of Atsena, said: “Receiving orphan designation from the EMA for both ATSN‑101 and ATSN‑201 underscores that these programs address significant unmet needs for patients who currently have no treatment options.”
He added: “With our pivotal trial for ATSN‑201 enrolling rapidly and our global pivotal trial for ATSN‑101 on track to begin later this year, we are closer than ever to delivering gene therapies that can change the trajectory of these inherited retinal diseases for patients in the U.S., Europe and beyond.”
Orphan designation in Europe is granted to therapies intended for rare, life‑threatening or chronically debilitating conditions and provides benefits such as reduced regulatory fees, protocol assistance and up to 10 years of market exclusivity. Both programmes have also received multiple designations from the U.S. Food and Drug Administration, including Orphan Drug and Fast Track.
Atsena dosed the first patient in the pivotal phase 3 cohort of the LIGHTHOUSE trial for ATSN‑201 in June 2026 and enrolment is progressing ahead of expectations. The study will include around 76 patients aged six and older, with microperimetry as the primary endpoint and a 52‑week readout.
A global pivotal phase 3 trial of ATSN‑101 is expected to begin later this year. The programme is being developed in collaboration with Nippon Shinyaku.
ATSN‑201 has already shown evidence of efficacy and safety in the completed phase 1/2 portion of LIGHTHOUSE, with most patients demonstrating improvements in retinal structure and visual function. Data now extend to two years in some participants and the therapy continues to show a favourable safety profile.
